This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.
Age: 18-80 years, no gender restriction;
Confirmed diagnosis of generalized myasthenia gravis (MG) with positive AchR or MuSK antibodies, meeting at least one of the following conditions#(1) Repetitive nerve stimulation suggesting neuromuscular transmission defect; (2) Positive response to neostigmine test; (3) Clinically judged improvement of --MG symptoms after oral cholinesterase inhibitor therapy;
Clinical classification of MG according to MGFA types IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa, IVb);
Baseline MG-ADL score ≥6, ocular-related score <50%;
Subjects positive for hepatitis B surface antigen (HBsAg) and/or hepatitis B core antibody (HBcAb) with detectable or quantifiable HBV DNA, positive for hepatitis C antibody (HCV Ab) with detectable or quantifiable HCV RNA, positive for HIV antibody, positive CMV DNA, or CMV DNA above the lower limit of detection; positive for syphilis antigen or antibody;
Presence of other uncontrolled active infections;
History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation;
Pregnant or breastfeeding women;
Shenzhen MagicRNA Biotechnology Co., Ltd
Lead sponsor
The First Affiliated Hospital of University of Science and Technology of China
Collaborator